It is hoped that patients treated with Miraxion at a dose of 2g per day (1g twice daily) for six months will demonstrate significant improvements in their motor functioning as measured by the Total Motor Score-4 (TMS-4) subscale of the Unified Huntington’s Disease Rating Scale (UHDRS).
Motor dysfunction is a prominent and progressively deteriorating feature of Huntington’s disease (HD), an ultimately fatal genetic disease causing uncontrolled movements, loss of intellectual faculties, emotional disturbances, psychosis and dementia.
Currently, there are limited treatment options for HD patients. There is no specifically approved treatment in the US to prevent the symptoms of HD or to delay its progression. Earlier studies of Miraxion showed encouraging improvements in motor function in these patients.
“Enrolling 300 individuals in this US and Canadian phase III trial is an enormous achievement for Amarin. With over 540 patients, the combined US, Canadian and European trials form one of the largest therapeutic programs ever conducted in HD,” said Rick Stewart, CEO of Amarin.
Miraxion has orphan drug designation in both the US and Europe and has fast-track designation from the FDA for review of the new drug application for HD.